Earlier this year, the US FDA approved Kalydeco for eight additional mutations (G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P or G1349D), adding another 400-or-so patients to the drug's ...
Launched in 2012, Vertex's Kalydeco has been a breakthrough in treating cystic fibrosis, being the first drug to target defective proteins which lead to damaging chest infections.